On February 20, 2024 PharmAbcine, Inc. ("PharmAbcine" or the "Company") (KOSDAQ: 208340), a clinical-stage public company developing next generation therapeutics to treat medical unmet needs, reported that on February 21st, 2023, PharmAbcine received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for PMC-403 for the treatment of a rare disease, Idiopathic Systemic Capillary Leak Syndrome (ISCLS) (Press release, PharmAbcine, FEB 20, 2024, View Source;bmode=view&idx=18106387&t=board [SID1234649177]).
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PMC-403 is a novel TIE2 binding and activating human IgG with the mechanism of stabilizing pathological and leaky blood vessels. TIE2 receptors, expressed on endothelial cells, are involved in vessel normalization processes such as angiogenesis and intercellular adhesion. PMC-403 activates the TIE2 receptor to improve vascular structure and stabilize abnormal blood vessels. This function has shown potential to be applicable for the treatment of various diseases caused by pathological vasculature.
The Company conducted research collaboration with Dr. Kirk Druey, Chief of the NIH’s Lung and Vascular Inflammation Section, to explore the efficacy of PMC-403 in ISCLS animal models. In this study, PMC-403 demonstrated efficacy in improving survival and alleviating vascular leakage in the mouse model of ISCLS induced by histamine. The study extends to the treatment of mice exposed to influenza, showing promising results in reducing vascular leakage.
ISCLS, or Clarkson Disease, is a severe systemic rare disorder characterized by the sudden and rapid exit of fluids and proteins from blood vessels into surrounding tissues due to the abnormal function of systemic vascular endothelial cells. This leads to symptoms such as hypotension, edema, and hypoalbuminemia, posing a significant threat with a 30% mortality rate upon acute onset.
ODD is a system that encourages the development of treatments for rare diseases or life-threatening diseases with fewer than 200,000 patients in the United States. Through this system, the development of orphan drugs is incentivized by various benefits such as subsidies, tax breaks, and market exclusivity due to the limited patient population, fostering rapid growth in this market. Additionally, the expedited review program for orphan drugs streamlines approval processes, incorporating FDA feedback and enabling faster development through smaller clinical trials.
According to Evaluate Pharma, the global orphan drug market is showing an average annual growth rate of 12%, from approximately $160 billion in 2021 to approximately $280 billion in 2026, and it is expected that orphan drug sales will account for 20% of the total prescription drug market by 2026.